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1、TheAAPSJournal2005;7(1)Article9(http://www.aapsj.org).DNA-basedTherapeuticsandDNADeliverySystems:AComprehensiveReviewSubmitted:February6,2004;Accepted:April8,2004;Published:April8,2005.SiddheshD.Patil,1DavidG.Rhodes,1andDianeJ.Burgess11DepartmentofPharmaceutic
2、alSciences,UniversityofConnecticut,Storrs,CT06269.ABSTRACTbasisofhumanlifebutalsoforthedevelopmentofanovelgroupoftherapeuticsmodeledonitsendogenousstructure.ThepastseveralyearshavewitnessedtheevolutionofgeneDNA-basedtherapeuticsincludeplasmidscontainingtrans-m
3、edicinefromanexperimentaltechnologyintoaviablegenesforgenetherapy,oligonucleotidesforantisenseandstrategyfordevelopingtherapeuticsforawiderangeofantigeneapplications,1ribozymes,DNAzymes,aptamers,humandisorders.NumerousprototypeDNA-basedbiophar-andsmallinterfer
4、ingRNAs(siRNAs).2,3Althoughmostofmaceuticalscannowcontroldiseaseprogressionbyinduc-tionand/orinhibitionofgenes.ThesepotenttherapeuticstheDNA-baseddrugsareinearlystagesofclinicaltrials,thisincludeplasmidscontainingtransgenes,oligonucleotides,classofcompoundshas
5、emergedinrecentyearstoyieldaptamers,ribozymes,DNAzymes,andsmallinterferingextremelypromisingcandidatesfordrugtherapyforawideRNAs.Althoughonly2DNA-basedpharmaceuticals(anrangeofdiseases,includingcancer,AIDS,neurologicaldis-antisenseoligonucleotideformulation,Vi
6、travene,(USA,orderssuchasParkinson’sdiseaseandAlzheimer’sdisease,1998),andanadenoviralgenetherapytreatment,Gendicineandcardiovasculardisorders.2,3(China,2003),havereceivedapprovalfromregulatoryElucidationofthehumangenomehasalsoprovidedamajoragencies;numerousca
7、ndidatesareinadvancedstagesofimpetusinidentifyinghumangenesimplicatedindiseases,humanclinicaltrials.SelectionofdrugsonthebasisofwhichmayeventuallyleadtothedevelopmentofDNA-DNAsequenceandstructurehasareducedpotentialfortox-baseddrugsforgenereplacementorpotentia
8、ltargetsforicity,shouldresultinfewersideeffects,andthereforegeneablation.4Inaddition,usinggenomicdata,potentDNA-shouldeventuallyyieldsaferdrugsthanthosecurrentlybaseddrugsmaybedeve
